Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome
editing company focused on the development of curative therapeutics
using CRISPR/Cas9 technology is presenting one oral presentation
and four poster presentations at the 27th Annual Congress of the
European Society of Gene and Cell Therapy (ESGCT) meeting taking
place October 22-25, 2019, in Barcelona, Spain.
“We are excited to share progress across Intellia’s in vivo and
ex vivo programs at this important scientific venue,” said Laura
Sepp-Lorenzino, Ph.D., chief scientific officer, Intellia
Therapeutics. “Our data shows the complexity of the edits we are
able to make with CRISPR/Cas9, while achieving important
therapeutically relevant results. We are building on the success of
our modular platform now having demonstrated consecutive targeted
knockout and insertion genome edits in preclinical studies.
Additionally, we presented data from our engineered cell therapy
program, which continues to demonstrate the use of CRISPR/Cas9 for
combined knockout and targeted integration in human T cells.”
Intellia Demonstrates Consecutive In Vivo Genome Editing
in Alpha-1 Antitrypsin Deficiency Mouse Model
Intellia’s oral presentation highlights its alpha-1 antitrypsin
deficiency (AATD) study showing that consecutive dosing of two
distinct lipid nanoparticle (LNP) formulations, in adult mice,
achieves two targeted genome editing events, resulting in knocking
out the faulty gene and restoring therapeutic levels of normal
alpha-1 antitrypsin protein (hAAT). Intellia’s approach for AATD
uses a modular hybrid delivery system combining a non-viral LNP
which encapsulates CRISPR/Cas9 with an adeno-associated virus (AAV)
carrying donor DNA template. Compared to traditional viral-based
delivery of gene editing components, Intellia’s LNP delivery system
can overcome the inherent limitations of immunogenicity to
facilitate multiple in vivo gene editing events.
In a mouse model harboring the human PiZ allele, the most severe
genetic defect in AATD patients, Intellia first reduced expression
of the defective protein using gene knockout. Three weeks following
the PiZ allele knockout, Intellia inserted the normal human alpha-1
antitrypsin gene, resulting in stable (throughout 12 weeks of
observation), therapeutically relevant circulating protein levels.
In the study, a sustained reduction of the circulating PiZ protein
levels of >98% was observed for over 15 weeks. This is the first
in vivo demonstration of a non-viral delivery platform, enabling a
consecutive dosing approach for achieving multiple genome edits in
the same tissue of the same animal. Intellia’s oral presentation,
titled “In Vivo Gene Knockout Followed by Targeted Gene Insertion
Results in Simultaneous Reduced Mutant Protein Levels and Durable
Transgene Expression,” will be given by Anthony Forget, Ph.D., on
October 25, 2019. This presentation will be available on
Intellia’s website at www.intelliatx.com.
Intellia’s Poster Presentations
WT1-Specific TCR Engineered Cell Therapy Studies
Intellia presented new in vitro data showing that
CRISPR/Cas9-mediated genome editing for in locus insertion,
combined with endogenous T Cell Receptor (TCR) knockout, leads to
significant reduction in mispairing of endogenous and transferred
TCR chains. This approach is expected to generate transgenic-TCR
(tg-TCR) T cell therapies for hematological cancers and solid
tumors. Results demonstrate a highly efficient reduction of >98%
in endogenous TCR α and β chains while reaching >70% insertion
rates of tg-TCRs without further purification. The poster titled
“Engineering of Highly Functional and Specific Transgenic T Cell
Receptor (TCR) T Cells Using CRISPR-Mediated In Locus Insertion
Combined with Endogenous TCR Knockout,” was presented on October
24, 2019, by Birgit Schultes, Ph.D.
Researchers also presented in vitro data showing that a library
of WT1-specific TCRs were generated, several of which Intellia is
currently evaluating as part of its lead engineered cell therapy
program targeting Acute Myeloid Leukemia (AML). This presentation,
“Generation of a Library of WT1-Specific T Cell Receptors (TCR) for
TCR Gene Edited T Cell Therapy of Acute Leukemia,” was presented on
October 23, 2019 by Intellia’s collaborator, Erica Carnevale,
Ph.D., IRCCS Ospedale San Raffaele.
Primary Hyperoxaluria Study
Intellia showed the continued progression of its modular
platform capability using CRISPR/Cas9 to knockout either
hydroxyacid oxidase 1 (Hao1) or lactate dehydrogenase A (Ldha),
leading to a dose-dependent and persistent reduction of urinary
oxalate levels in a Primary Hyperoxaluria Type 1 (PH1) mouse model.
Data shows Ldha gene disruption also decreased LDH enzyme activity
in the liver and did not impair the disposition of lactate in
either wild type or renally-impaired mice. These results highlight
the potential of editing genes in the glyoxylate detoxification
pathway using a non-viral delivery approach as a one-time treatment
option for PH1. These data were presented as a poster, titled
“CRISPR/Cas9-Mediated Gene Knockout to Address Primary
Hyperoxaluria,” by Sean Burns, M.D., on October 24,
2019.
Off-Target Screening Platform
Intellia demonstrated its approach to assess off-target activity
to identify highly specific CRISPR/Cas9 guides. Results from
targeted off-target sequencing in edited cells showed that
biochemical off-target discovery approaches were the most sensitive
and accurate. These data were presented as a poster on October 23,
2019, titled “In Silico, Biochemical and Cell-Based Integrative
Genomics Identifies Precise CRISPR/Cas9 Targets for Human
Therapeutics,” by Dan O’Connell, Ph.D.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on developing proprietary, curative therapeutics using the
CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology
has the potential to transform medicine by permanently editing
disease-associated genes in the human body with a single treatment
course, and through improved cell therapies that can treat cancer
and immunological diseases, or can replace patients’ diseased
cells. The combination of deep scientific, technical and clinical
development experience, along with its leading intellectual
property portfolio, puts Intellia in a unique position to unlock
broad therapeutic applications of the CRISPR/Cas9 technology and
create a new class of therapeutic products. Learn more about
Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow
us on Twitter @intelliatweets.
Forward-Looking Statements
This press release contains “forward-looking statements”
of Intellia Therapeutics, Inc. (“Intellia” or the
“Company”) within the meaning of the Private Securities Litigation
Reform Act of 1995. These forward-looking statements include, but
are not limited to, express or implied statements regarding
Intellia’s beliefs and expectations regarding its planned
submission of an IND application for NTLA-2001 in mid-2020; its
plans to generate preclinical and other data necessary to nominate
a first engineered cell therapy development candidate for its AML
program by the end of 2019; its plans to advance and complete
preclinical studies, including non-human primate studies for its
ATTR program, AML program and other in vivo and ex
vivo programs such as its AATD program; develop our
proprietary LNP-AAV hybrid delivery system to advance our complex
genome editing capabilities, such as gene insertion; its
presentation of additional data at upcoming scientific conferences
regarding CRISPR-mediated, targeted transgene insertion in the
liver of NHPs, using F9 as a model gene, via the Company’s
proprietary LNP-AAV delivery technology, and other preclinical data
by the end of 2019; the advancement and expansion of its
CRISPR/Cas9 technology to develop human therapeutic products, as
well as maintain and expand its related intellectual property
portfolio; the ability to demonstrate its platform’s modularity and
replicate or apply results achieved in preclinical studies,
including those in its ATTR and AML programs, in any future
studies, including human clinical trials; its ability to develop
other in vivo or ex vivo cell therapeutics of
all types, and those targeting WT1 in AML in particular, using
CRISPR/Cas9 technology; the impact of its collaborations on its
development programs, including but not limited to its
collaboration with Regeneron Pharmaceuticals, Inc. or Ospedale
San Raffaele; statements regarding the timing of regulatory filings
regarding its development programs; and the ability to fund
operations into the second half of 2021.
Any forward-looking statements in this press release are based
on management’s current expectations and beliefs of future events,
and are subject to a number of risks and uncertainties that could
cause actual results to differ materially and adversely from those
set forth in or implied by such forward-looking statements. These
risks and uncertainties include, but are not limited to: risks
related to Intellia’s ability to protect and maintain our
intellectual property position, including through our arbitration
proceedings against Caribou; risks related to Intellia’s
relationship with third parties, including our licensors; risks
related to the ability of our licensors to protect and maintain
their intellectual property position; uncertainties related to the
initiation and conduct of studies and other development
requirements for our product candidates; the risk that any one or
more of Intellia’s product candidates will not be successfully
developed and commercialized; the risk that the results of
preclinical studies will not be predictive of future results in
connection with future studies; and the risk that Intellia’s
collaborations with Novartis or Regeneron or its
other ex vivo collaborations will not continue or will
not be successful. For a discussion of these and other risks and
uncertainties, and other important factors, any of which could
cause Intellia’s actual results to differ from those contained in
the forward-looking statements, see the section entitled “Risk
Factors” in Intellia’s most recent annual report on Form 10-K as
well as discussions of potential risks, uncertainties, and other
important factors in Intellia’s other filings with
the Securities and Exchange Commission. All information in
this press release is as of the date of the release,
and Intellia undertakes no duty to update this information
unless required by law.
Intellia Contacts:
Media: Jennifer Mound Smoter Senior Vice
President External Affairs & Communications +1 857-706-1071
jenn.smoter@intelliatx.com
Investors: Lina Li Associate Director Investor
Relations +1 857-706-1612 lina.li@intelliatx.com
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